Public Release: 

New treatment offers hope for children with debilitating skin and muscle disease

Patients who have failed multiple drug treatments show significant improvements

European League Against Rheumatism

London, United Kingdom, 10 June 2016: The results of a UK study presented today at the European League Against Rheumatism Annual Congress (EULAR 2016) showed that tumour necrosis factor inhibitor (anti-TNF) treatment is effective at improving both muscle and skin involvement in children with juvenile dermatomyositis (JDM). These findings bring new hope to JDM patients who have failed to respond to multiple drug treatments and who, as a result, have a greater risk of painful complications and premature death.

JDM is a rare chronic autoimmune disease of children characterised by inflammation of the muscles, skin and other organs. , In the UK, the reported incidence of JDM is two to three cases per million children younger than 16 years old, with a median age at onset of 6.8 years; JDM is more common in girls than boys, by a ratio of approximately 2.3:1. Characteristic findings include skin rash, skin ulceration, and muscle weakness.2,3

In general, children with JDM are able to lead normal lives; however, some patients with JDM have a disease course that is refractory to multiple drug treatments. For those children where treatments have failed, prolonged disease activity has been shown to be associated with increased mortality and complications, such as scarring, pain due to trapped nerves, and shortening of the muscles causing joints to stay bent.

"High levels of the cell signalling protein TNF have been reported in JDM patients with a long disease course, suggesting this immune cell regulator may play a significant role in refractory disease," said Dr Raquel Campanilho-Marques of the Institute of Child Health, University College London, UK. "There are no published clinical trials (only case reports) of this therapy, but some are in progress. Our study is one of the largest to explore the efficacy and safety of anti-TNF therapy in a large independent cohort of JDM patients."

Evaluation of 66 patients with JDM, recruited from the UK JDM Cohort and Biomarker Study and actively treated with anti-TNF agents, showed significant improvements in muscle and skin involvement, as well as in overall disease activity. There were significant changes in the median values of two standard muscle measurements: the Childhood Myositis Assessment Scale and Manual Muscle Testing (p<0.0001 and p=0.0097 respectively). There were also significant improvements in skin involvement assessed using the modified skin Disease Activity Score (p<0.0001). Global disease activity also improved significantly (p<0.0001).

Around one quarter of the patients in this study switched their anti-TNF treatment, with just under two-thirds of these switches due to therapy failure, one quarter due to adverse events and one eighth due to patient preference for subcutaneous administration. Of 21 adverse reactions registered, seven were considered severe (anaphylactic reactions on infliximab infusion). Three quarters of the mild to moderate adverse reactions were due to infection; in four of which, the patient was switched to another TNF antagonist, while in the remaining patients, temporarily withholding the drug proved sufficient. No cases of TB were registered.

Abstract Number: OP0221

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NOTES TO EDITORS:

For further information on this study, or to request an interview with the study lead, please do not hesitate to contact the EULAR congress Press Office in the London
Suite at ExCel London during EULAR 2016 or on:
Email: eularpressoffice@cohnwolfe.com
Onsite tel: +44 (0) 7725 915 492 / +44 (0) 7786 171 476
Twitter: @EULAR_Press
Youtube: Eular Pressoffice

About EULAR

The European League Against Rheumatism (EULAR) is an umbrella organisation which represents scientific societies, health professional associations and organisations for people with Rheumatic Musculoskeletal Diseases (RMD) throughout Europe.

EULAR aims to promote, stimulate and support the research, prevention, and treatment of RMD and the rehabilitation of those it affects.

EULAR underlines the importance of combating rheumatic diseases not only by medical means, but also through a wider context of care for rheumatic patients and a thorough understanding of their social and other needs. EULAR is supported in this mission by its 45 scientific member societies, 36 PARE (People with Arthritis/Rheumatism in Europe) organisations, 22 HPR (Health Professionals in Rheumatology) associations and 23 corporate members.

The EULAR Annual European Congress of Rheumatology is the foremost international medical meeting announcing the latest research on rheumatic and musculoskeletal diseases. EULAR 2016 is expected to attract over 14'000 delegates from around 120 countries. Most if not all professions working in the vast field of RMD will be represented.

To find out more about the activities of EULAR, visit: http://www.eular.org

References

1. EULAR 2016; London: Abstract OP0221

2. Bohan A, Peter JB. Polymyositis and dermatomyositis (first of two parts). N Engl J Med. 1975; 292 (7): 344-7

3. Bohan A, Peter JB. Polymyositis and dermatomyositis (second of two parts). N Engl J Med. 1975; 292 (8): 403-7

4. Wedderburn LR, Rider LG. Juvenile Dermatomyositis: New Developments in Pathogenesis, Assessment and Treatment. Best practice & research Clinical rheumatology. 2009; 23(5): 665-678

5. Symmons DP, Sills JA, Davis SM. The incidence of juvenile dermatomyositis: results from a nation-wide study. Br J Rheumatol. 1995 ; 34(8): 732-6

6. McCann LJ, Juggins AD, Maillard SM, et al, Juvenile Dermatomyositis Research Group. The Juvenile Dermatomyositis National Registry and Repository (UK and Ireland)-clinical characteristics of children recruited within the first 5 yr. Rheumatology (Oxford). 2006; 4(10): 1255-60

7. Reed AM. Juvenile dermatomyositis http://emedicine.medscape.com/article/1417215-overview [Accessed 5 May 2016]

8. Pachman LM, Liotta-Davis MR, Hong DK et al. TNFalpha-308A allele in juvenile dermatomyositis: association with increased production of tumor necrosis factor alpha, disease duration, and pathologic calcifications. Arthritis Rheum 2000; 43: 2368-77

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